Objective: To evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation with high-intensity immunosuppresion in patients with multiple sclerosis (MS).

Methods: A Phase II clinical trial of AHSCT using a BEAM + ATG chemotherapeutic regimen for patients with relapsing remitting and secondary progressive MS began at St Vincent's Hospital in December 2010. Patients eligible for AHSCT had an Expanded Disability Status Scale [EDSS] of 3.0-6.5, have failed two prior disease-modifying therapies and display evidence of ongoing disease activity with clinical relapses and/or new MRI lesions prior to AHSCT.

Conclusion: AHSCT was effective for inducing long-term sustained remissions in the majority of active RRMS patients at 5 years. Younger age and lower baseline EDSS scores were associated with better outcomes. The results support the need for further refinement of which patients are most suitable to undergo AHSCT and for longer follow up of cohorts.